Treatment algorithm

Please note that formulations/routes and doses may differ between drug names and brands, drug formularies, or locations. Treatment recommendations are specific to patient groups: see disclaimer

ONGOING

attenuated WAS

1st line – observation

Back
ONGOING
|
attenuated WAS
1st line – 

observation

If clinically well, no treatment is required. Patients should be monitored for development of eczema or symptomatic thrombocytopenia (i.e., worsening bruising or any spontaneous bleeding). Frequency and severity of infections should be monitored, as well as development of any form of autoimmunity (especially autoimmune cytopenias). Development of haematological malignancies, particularly lymphoma, is uncommon but should be considered.

Children with attenuated WAS should be vaccinated according to the national childhood vaccination programme.

Consider – prophylactic antibiotics

Back
ONGOING
|
attenuated WAS
|
with frequent minor infections or any severe infections
Consider – 

prophylactic antibiotics

Additional treatment recommended for SOME patients in selected patient group

Prophylactic antibiotics should be started. Trimethoprim/sulfamethoxazole is the antibiotic of choice; however, azithromycin may be used if this is contraindicated.

Primary options

trimethoprim/sulfamethoxazole: children >2 months of age: 2.5 mg/kg orally twice daily; adults: 160 mg orally twice daily

More

OR

azithromycin: children and adults: 10 mg/kg orally once daily for 3 consecutive days every 14 days, maximum 500 mg/day

Consider – intravenous or subcutaneous immunoglobulin

Back
ONGOING
|
attenuated WAS
|
with frequent minor infections or any severe infections
Consider – 

intravenous or subcutaneous immunoglobulin

Additional treatment recommended for SOME patients in selected patient group

If symptoms persist/worsen or if specific antibody production is abnormal (as indicated by vaccine responses), immunoglobulin replacement is indicated and the patient's classification as attenuated WAS should be reviewed.

Subcutaneous administration of immunoglobulin is very well tolerated even in the presence of thrombocytopenia, and facilitates care at home.

Primary options

normal immunoglobulin human: children and adults: consult specialist for guidance on dose

Consider – step-wise treatment with emollients, topical corticosteroids, oral corticosteroids, or topical tacrolimus

Back
ONGOING
|
attenuated WAS
|
with eczema
Consider – 

step-wise treatment with emollients, topical corticosteroids, oral corticosteroids, or topical tacrolimus

Additional treatment recommended for SOME patients in selected patient group

Emollients (e.g., liquid paraffin) are tried first. Bath emollients and a soap substitute are also recommended.

If further treatment is required, topical corticosteroids can be added. The strength will depend on eczema severity.

If not controlled by topical therapy, systemic corticosteroids can be added.

Topical tacrolimus can be used as a corticosteroid-sparing agent, although potential adverse effects should be fully discussed.

Primary options

emollient topical: children and adults: apply to the affected area(s) when required, particularly after bathing

Secondary options

hydrocortisone topical: children and adults: (1%) apply sparingly to the affected area(s) twice daily

OR

betamethasone valerate topical: children and adults: (0.1%) apply sparingly to the affected area(s) twice daily

OR

fluocinolone topical: children >3 months of age and adults: (0.025%) apply sparingly to the affected area(s) twice daily

OR

clobetasol topical: children >12 years of age and adults: (0.05%) apply sparingly to the affected area(s) twice daily

Tertiary options

prednisolone: children and adults: 1 mg/kg/day orally given in 2 divided doses for 2-3 weeks, then taper gradually

OR

tacrolimus topical: children >2 years of age: (0.03%) apply sparingly to the affected area(s) once or twice daily; children >15 years of age and adults: (0.03% or 0.1%) apply sparingly to the affected area(s) once or twice daily

Consider – antifibrinolytic treatment

Back
ONGOING
|
attenuated WAS
|
with bruising
Consider – 

antifibrinolytic treatment

Additional treatment recommended for SOME patients in selected patient group

Aminocaproic acid is used for acute bleeding and is continued until bleeding is controlled. Consider if the patient has thrombocytopenia with moderate bruising or for bruising following trauma.

Can be used for a period of several days to reduce bruising after a specific trauma or trialled as continuous treatment to reduce easy bruising.

Primary options

aminocaproic acid: children: 100-200 mg/kg orally as a loading dose, followed by 100 mg/kg every 4-6 hours; adults: 4-5 g orally as a loading dose, followed by 1 g/hour for 8 hours, maximum 30 g/day

Consider – thrombocytopenia treatment

Back
ONGOING
|
attenuated WAS
|
with bleeding
Consider – 

thrombocytopenia treatment

Additional treatment recommended for SOME patients in selected patient group

Platelet transfusion is first-line treatment. Check platelet count before and after transfusion to document increment. If significant drop in initial platelet count from patient's usual level, consider autoimmune process and measure antiplatelet antibodies. Blood products should be irradiated and should be cytomegalovirus negative.

If poor increment post transfusion or suspicion of autoimmune process, use HLA-identical platelets, and treat with systemic corticosteroids plus high-dose immunoglobulin plus rituximab. Prior to commencement of immunoglobulin treatment, save serum for detection of antiplatelet antibodies.

Primary options

prednisolone: children and adults: 2 mg/kg/day orally for 1-2 weeks then taper gradually, maximum 60 mg/day

or

methylprednisolone: children and adults: 4 mg/kg/day orally/intravenously for 1-2 weeks then taper gradually, maximum 60 mg/day

-- AND --

normal immunoglobulin human: children and adults: consult specialist for guidance on dose

-- AND --

rituximab: children and adults: consult specialist for guidance on dose

Consider – splenectomy

Back
ONGOING
|
attenuated WAS
|
with bleeding
Consider – 

splenectomy

Additional treatment recommended for SOME patients in selected patient group

Elective splenectomy can be considered for management of severe thrombocytopenia (in the absence of immunodeficiency and autoimmunity).[19] It is highly effective at correcting platelet counts. However, there is an increased risk of sepsis unless precautions are taken. If not on immunoglobulin these include pre-splenectomy immunisation againstStreptococcus pneumoniae,Haemophilus influenzae type b, andNeisseria meningitidis. CDC Advisory Committee for Immunization Practices: recommendations Opens in new window Consider re-immunisation on a 5-year basis or according to blood antibody levels (which should be serially monitored so that boosters are given when levels are low). In addition, patients will require lifelong penicillin prophylaxis, and appropriate malaria prophylaxis should be taken where necessary if travelling.

Consider – immunosuppression therapy

Back
ONGOING
|
attenuated WAS
|
with autoimmunity
Consider – 

immunosuppression therapy

Additional treatment recommended for SOME patients in selected patient group

Tailor to type of autoimmunity. Rituximab is effective for autoimmune cytopenias (such as haemolytic anaemia, thrombocytopenia, or neutropenia). Otherwise, treatment is specific to the type of autoimmunity. A multidisciplinary approach may be required (e.g., involving rheumatology or gastroenterology teams for arthritis/colitis, respectively).

The patient should be reclassified as severe WAS.

Consider – consultant referral

Back
ONGOING
|
attenuated WAS
|
with malignancy
Consider – 

consultant referral

Additional treatment recommended for SOME patients in selected patient group

Assessment and treatment should be guided by a paediatric haematologist/oncologist. Treatment depends on pathology.

The patient should be reclassified as severe WAS.

severe, classical WAS

1st line – prophylactic antibiotics

Back
ONGOING
|
severe, classical WAS
1st line – 

prophylactic antibiotics

For all patients with severe, classical WAS prophylactic antibiotics are recommended until successfully transplanted.

Primary options

trimethoprim/sulfamethoxazole: children >2 months of age: 2.5 mg/kg orally twice daily; adults: 160 mg orally twice daily

More

OR

azithromycin: children and adults: 10 mg/kg orally once daily for 3 consecutive days every 14 days, maximum 500 mg/day

Consider – vaccination

Back
ONGOING
|
severe, classical WAS
Consider – 

vaccination

Additional treatment recommended for SOME patients in selected patient group

Pneumococcal vaccine is recommended for all children <2 years, and for unimmunised children between 24 and 59 months old who are at high risk for pneumococcal infections, unless they are on immunoglobulin therapy. Patients with severe WAS should not receive live vaccinations but should receive the inactivated influenza vaccination annually, as well as coronavirus disease 2019 (COVID-19) vaccinations: local guidance should be followed for patients who are moderately to severely immunocompromised.

Consider – intravenous or subcutaneous immunoglobulin

Back
ONGOING
|
severe, classical WAS
Consider – 

intravenous or subcutaneous immunoglobulin

Additional treatment recommended for SOME patients in selected patient group

If ongoing infection problems despite prophylactic antibiotics, commence immunoglobulin replacement and reconsider whether patient has classical WAS.

Subcutaneous administration of immunoglobulin is very well tolerated even in the presence of thrombocytopenia, and facilitates care at home.

Primary options

normal immunoglobulin human: children and adults: consult specialist for guidance on dose

Plus – haematopoietic stem cell transplant (HSCT)

Back
ONGOING
|
severe, classical WAS
|
with haematopoietic stem cell match
Plus – 

haematopoietic stem cell transplant (HSCT)

Treatment recommended for ALL patients in selected patient group

Should be performed where a matched sibling or matched unrelated donor is available. Outcomes for HSCT are excellent, with mean overall survival of >90% according to one report.[22]

If a donor is found, continue prophylactic antibiotics and immunoglobulins until successfully transplanted.

Necessitates penicillin prophylaxis for life.

Consider – splenectomy

Back
ONGOING
|
severe, classical WAS
|
no haematopoietic stem cell match
Consider – 

splenectomy

Additional treatment recommended for SOME patients in selected patient group

If no match can be found, consider splenectomy if platelets <10 x 10⁹/L or if history of significant bleeding episode.

There is an increased risk of sepsis unless precautions are taken. If not on immunoglobulin these include pre-splenectomy immunisation againstStreptococcus pneumoniae,Haemophilus influenzae type b, andNeisseria meningitidis. CDC Advisory Committee for Immunization Practices: recommendations Opens in new window Consider re-immunisation on a 5-year basis or according to blood antibody levels (which should be serially monitored so that boosters are given when levels are low). In addition, patients will require lifelong penicillin prophylaxis, and appropriate malaria prophylaxis should be taken where necessary if travelling.

Consider – step-wise treatment starting with emollients

Back
ONGOING
|
severe, classical WAS
|
with eczema
Consider – 

step-wise treatment starting with emollients

Additional treatment recommended for SOME patients in selected patient group

Treatment is carried out in a step-wise approach, starting with emollients and progressing to topical corticosteroids and topical calcineurin inhibitors, as required, for the control of symptoms.

See Eczema.

Consider – antifibrinolytic treatment

Back
ONGOING
|
severe, classical WAS
|
with bruising
Consider – 

antifibrinolytic treatment

Additional treatment recommended for SOME patients in selected patient group

Aminocaproic acid is given for acute bleeding until bleeding is controlled. Consider if platelets <10 x 10⁹/L with moderate bruising or for bruising following trauma.

Can be used for a period of several days to reduce bruising after a specific trauma or trialled as continuous treatment to reduce easy bruising.

Primary options

aminocaproic acid: children: 100-200 mg/kg orally as a loading dose, followed by 100 mg/kg every 4-6 hours; adults: 4-5 g orally as a loading dose, followed by 1 g/hour for 8 hours, maximum 30 g/day

Consider – thrombocytopenia treatment

Back
ONGOING
|
severe, classical WAS
|
with bleeding
Consider – 

thrombocytopenia treatment

Additional treatment recommended for SOME patients in selected patient group

Platelet transfusion is first-line treatment. Platelet count should be checked before and after transfusion to document increment. If significant drop in initial platelet count from patient's usual level, consider autoimmune process and measure antiplatelet antibodies. Blood products should be irradiated and should be cytomegalovirus negative.

If poor increment post transfusion or suspicion of autoimmune process, use HLA-identical platelets, and treat with systemic corticosteroids plus high-dose immunoglobulin plus rituximab. Prior to commencement of immunoglobulin treatment, save serum for detection of antiplatelet antibodies.

If intractable bleeding despite treatment, or if significant bleeding episode and low possibility of urgent bone marrow transplantation, consider splenectomy.

With splenectomy there is an increased risk of sepsis unless precautions are taken. If not on immunoglobulin these include pre-splenectomy immunisation againstStreptococcus pneumoniae,Haemophilus influenzae type b, andNeisseria meningitidis. CDC Advisory Committee for Immunization Practices: recommendations Opens in new window Consider re-immunisation on a 5-year basis or according to blood antibody levels (which should be serially monitored so that boosters are given when levels are low). In addition, patients will require lifelong penicillin prophylaxis, and appropriate malaria prophylaxis should be taken where necessary if travelling.

Primary options

prednisolone: children and adults: 2 mg/kg/day orally for 1-2 weeks then taper gradually, maximum 60 mg/day

or

methylprednisolone: children and adults: 4 mg/kg/day orally/intravenously for 1-2 weeks then taper gradually, maximum 60 mg/day

-- AND --

normal immunoglobulin human: children and adults: consult specialist for guidance on dose

-- AND --

rituximab: children and adults: consult specialist for guidance on dose

Consider – immunosuppression therapy

Back
ONGOING
|
severe, classical WAS
|
with autoimmunity
Consider – 

immunosuppression therapy

Additional treatment recommended for SOME patients in selected patient group

Tailor to type of autoimmunity. Rituximab is effective for autoimmune cytopenias (such as haemolytic anaemia, thrombocytopenia, or neutropenia). Otherwise, treatment is specific to the type of autoimmunity. A multidisciplinary approach may be required (e.g., involving rheumatology or gastroenterology teams for arthritis/colitis, respectively).

Consider – consultant referral

Back
ONGOING
|
severe, classical WAS
|
with malignancy
Consider – 

consultant referral

Additional treatment recommended for SOME patients in selected patient group

Assessment and treatment should be guided by a paediatric haematologist/oncologist. Treatment depends on pathology.

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Choose a patient group to see our recommendations

Please note that formulations/routes and doses may differ between drug names and brands, drug formularies, or locations. Treatment recommendations are specific to patient groups. See disclaimer

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